Sevasemten(Sevasemten) - 药物靶点：MYH4_在研适应症：贝克型肌营养不良，糖原贮积病Ⅴ型，肢带型肌营养不良_专利_临床

概要

基本信息

药物类型

小分子化药

别名

type II fast skeletal myosin inhibitor (Edgewise Therapeutics)、EDG 5506、EDG-5506

+ [1]

靶点

MYH4

作用方式

抑制剂

作用机制

MYH4抑制剂(myosin heavy chain 4 inhibitors)

治疗领域

神经系统疾病遗传病与畸形皮肤和肌肉骨骼疾病+ [2]

在研适应症

贝克型肌营养不良糖原贮积病Ⅴ型肢带型肌营养不良+ [1]

非在研适应症-

原研机构

Edgewise Therapeutics, Inc.

在研机构

Edgewise Therapeutics, Inc.

非在研机构-

权益机构

Edgewise Therapeutics, Inc.Servier SAS

最高研发阶段临床3期

首次获批日期-

最高研发阶段(中国)-

特殊审评孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、快速通道 (美国)

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结构/序列

分子式C16H11F4N5O2

InChIKeyXFPJKQRXBAFXNK-UHFFFAOYSA-N

CAS号2417395-15-2

关联

10

项与 Sevasemten 相关的临床试验

NCT06916897

/ Completed临床1期

A Phase 1, Open-Label Study to Evaluate the Effect of Verapamil and Food on the Pharmacokinetics of Sevasemten in Healthy Adult Subjects

The purposes of this Phase 1 study of sevasemten are to:
1. Evaluate the effect of multiple-dose administration of verapamil on the single-dose of sevasemten in healthy adults
2. Evaluate the safety and tolerability of a single dose of sevasemten administered with and without verapamil in healthy adult subjects.
3. Evaluate the safety and tolerability of a single dose of sevasemten administered with and without food in healthy adult subjects.

开始日期2025-01-08

申办/合作机构

Edgewise Therapeutics, Inc.

NCT06100887

/ Active, not recruiting临床2期

A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety, Pharmacokinetics, and Biomarkers in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.

开始日期2024-03-22

申办/合作机构

Edgewise Therapeutics, Inc.

NCT06066580

/ Enrolling by invitation临床2期

An Open-Label Extension Study to Assess the Long-term Effect of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy

开始日期2023-11-02

申办/合作机构

Edgewise Therapeutics, Inc.

[+1]

100 项与 Sevasemten 相关的临床结果

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100 项与 Sevasemten 相关的转化医学

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100 项与 Sevasemten 相关的专利（医药）

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2

项与 Sevasemten 相关的文献（医药）

2023-05-15·The Journal of clinical investigation

Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy

Article

作者: Peter, Angela K ; Qian, Ying ; Sweeney, H Lee ; Patsalos, Andreas ; Newell-Stamper, Breanne L ; Rodriguez, Hector M ; Brooks, Susan V ; Vera, Carlos ; Nagy, Laszlo ; Juehne, Twlya I ; Lehman, Sarah ; Madden, Molly ; Rutledge, Alexis ; Hunt, Kevin ; Barthel, Ben ; Yu, Jinsheng ; Schlachter, Stephen ; Van Deusen, Ashleigh ; Leinwand, Leslie A ; Russell, Alan J ; Nghiem, Peter ; Su, Yu ; Luo, Yangyi E ; Claflin, Dennis R ; DuVall, Mike ; Barton, Elisabeth R ; Koch, Kevin ; Robertson, Ben

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by absence of the protein dystrophin, which acts as a structural link between the basal lamina and contractile machinery to stabilize muscle membranes in response to mechanical stress. In DMD, mechanical stress leads to exaggerated membrane injury and fiber breakdown, with fast fibers being the most susceptible to damage. A major contributor to this injury is muscle contraction, controlled by the motor protein myosin. However, how muscle contraction and fast muscle fiber damage contribute to the pathophysiology of DMD has not been well characterized. We explored the role of fast skeletal muscle contraction in DMD with a potentially novel, selective, orally active inhibitor of fast skeletal muscle myosin, EDG-5506. Surprisingly, even modest decreases of contraction (<15%) were sufficient to protect skeletal muscles in dystrophic mdx mice from stress injury. Longer-term treatment also decreased muscle fibrosis in key disease-implicated tissues. Importantly, therapeutic levels of myosin inhibition with EDG-5506 did not detrimentally affect strength or coordination. Finally, in dystrophic dogs, EDG-5506 reversibly reduced circulating muscle injury biomarkers and increased habitual activity. This unexpected biology may represent an important alternative treatment strategy for Duchenne and related myopathies.

MUSCLE & NERVE

A Phase 1, Double‐Blind, Placebo‐Controlled Trial of Sevasemten (EDG‐5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy

Article

作者: Kilburn, Nicole Rempel ; Koch, Kevin ; Bronson, Abby ; Silverman, Jeffrey A. ; Gordon, Gilad ; MacDougall, James ; Barthel, Ben ; Madden, Molly ; Russell, Alan J ; DuVall, Michael ; Donovan, Joanne ; Evanchik, Marc

ABSTRACT:

Introduction/Aims:

Sevasemten (EDG‐5506) is an orally administered, investigational small molecule that selectively modulates fast muscle fiber contraction by inhibiting fast myosin ATPase. This study assessed the safety, tolerability, and pharmacokinetics (PK)/pharmacodynamics (PD) of sevasemten in healthy adult volunteers (HVs) and adults with Becker muscular dystrophy (BMD).

Methods:

This randomized, double‐blind, placebo‐controlled phase 1 study was conducted at a single site. Eligible participants were 18–55 years of age with a body mass index < 30 kg/m2; adults with BMD were required to have a confirmed diagnosis based on documentation of pathogenic variant(s) in the dystrophin gene and a BMD phenotype. Participants were randomized 3:1 to receive sevasemten or placebo for up to 14 days. Endpoints included adverse events (AEs), sevasemten PK and muscle concentration, and changes in biomarkers of muscle injury.

Results:

The study enrolled 97 HVs in 7 single‐dose cohorts (N = 57) and 5 multiple‐dose cohorts (N = 40). Seven adults with BMD were enrolled in a multiple‐dose cohort. There were no serious AEs or AEs leading to trial discontinuation; the most common AEs were dizziness and somnolence. For adults with BMD, serum biomarkers of muscle injury trended down progressively with sevasemten treatment (average maximal reductions of 70% for creatine kinase, 98% for fast skeletal muscle troponin I, and 45% for myoglobin). Plasma proteomic analysis identified a signature of 125 elevated proteins characteristic of BMD that was reversibly lowered with sevasemten treatment.

Discussion:

Sevasemten was generally well tolerated. Preliminary observations of decreases in biomarkers of muscle damage in adults with BMD support further clinical development.Trial Registration: NCT04585464

117

项与 Sevasemten 相关的新闻（医药）

2026-06-02

·GBIHealth

逾30亿美元，海思科与礼来达成授权与研发合作

企业动态 No.1 /先声药业获普祺医药JAK抑制剂凝胶独家推广权益 2026年6月1日，先声药业（2096.HK）宣布，与北京普祺医药科技股份有限公司（以下简称“普祺医药”）就普美昔替尼凝胶订立独家推广服务协议。普美昔替尼凝胶是普祺医药研发的JAK抑制剂，其成人与青少年特应性皮炎适应证新药上市申请（NDA）已获国家药品监督管理局药品审评中心（CDE）受理。根据协议，先声药业将获得普美昔替尼凝胶在中国内地、香港、澳门所有皮肤病领域适应证的独家推广权益。 ->点击文末阅读原文，解锁完整双语新闻 No.2 / 逾30亿美元，海思科与礼来达成授权与研发合作 2026年6月2日，海思科（002653.SZ）发布公告称，公司与礼来（NYSE：LLY）签署《授权与研发合作协议》，双方将在多个疾病领域的创新药物研发开展战略合作。根据协议，公司将负责礼来选定的最多五个靶点创新药项目的发现及早期研发工作。礼来将获得相关项目的全球独家权利或除中国大陆和港澳台以外的全球独家权利，公司则保留部分项目在中国大陆和港澳台的独家权利。根据协议约定，公司有权获得最高8700万美元的首付款和近期付款，最高29.67亿美元的后续里程碑付款，并有权根据产品未来净销售额获得分级销售提成。此次合作是公司与礼来之间首次达成的授权与研发合作，双方将依托各自在创新药开发的优势，共同加速创新药物的全球推进。 ->点击文末阅读原文，解锁完整双语新闻 No.3 / 施维雅收购Edgewise Therapeutics肌营养不良业务 2026年6月1日，施维雅宣布，将收购Edgewise Therapeutics Inc.（Nasdaq：EWTX）的肌营养不良症业务，总金额最高可达26.5亿美元，包括15.5亿美元的首付款，以及最高可达11亿美元的监管和商业里程碑付款。目前，该交易已获得两家公司各自管理机构的批准，尚需获得监管部门的批准并满足惯例成交条件，预计将于2026年第三季度完成交割。 Edgewise 是一家专注于为严重肌肉疾病开发创新疗法的生物制药公司。此次对 Edgewise Therapeutics 肌营养不良症业务的收购，涵盖了其相关研发技术能力以及sevasemten。Sevasemten 是一款在研的口服、同类首创（First-in-class）快肌肌球蛋白抑制剂，旨在保护罕见肌营养不良症患者不稳定的肌肉，使其免受收缩诱导的损伤。目前，sevasemten正在进行针对贝克型肌营养不良症（BMD）的关键队列研究，以及针对杜氏肌营养不良症（DMD）的Ⅱ期临床研究。 ->点击文末阅读原文，解锁完整双语新闻行业政策 No.1 / 药监局发文调整医疗器械分类 2026年6月1日，国家药监局发布《关于医疗器械分类调整有关工作的公告（2026年第52号）》和《医疗器械分类目录动态调整工作程序的公告（2026年第53号）》，新版分类调整公告细化了不同调整情形相关工作要求，明确了注册人、备案人和相关生产企业履行主体责任要求。新版动态调整工作程序增加《体外诊断试剂分类规则》作为动态调整法规依据，增加《体外诊断试剂注册与备案管理办法》作为动态调整后注册备案事项的法规依据，将《第一类医疗器械产品目录》《体外诊断试剂分类目录》的动态调整纳入适用目录范围。公告适用于具体医疗器械或者品类整体调整的要求，对于此类调整国家药监局会以公告等形式对外公开。医疗器械相关分类目录调整涉及产品管理属性或者管理类别调整时是否设置产品注册/备案过渡期以及过渡期设置时限，是在综合考虑产品风险、产业发展、临床应用需求等因素和各方意见后确定的。《动态调整工作程序》第十条（一）明确了不同调整情形的过渡期设置原则，规定“由不作为医疗器械管理调整为作为第二类或者第三类医疗器械管理的，或者由低类别医疗器械调整为高类别的，一般设置2~3年过渡期。其中，对于不需开展临床试验的，产品注册过渡期一般设置为2年。对于需开展临床试验的，产品注册过渡期一般设置为3年”。同时，充分考虑了各种特殊情形，在《动态调整工作程序》第十条（二）明确“可以设置3年以上过渡期，原则上过渡期最长不超过5年”。 ->点击文末阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I ” 自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作 ▶ olivia.xu@generalbiologic.com 数据库 | 咨询服务 | 资讯追踪 ▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

2026-06-01

·Medaverse

26.5亿美元！施维雅收购Edgewise Therapeutics肌肉萎缩症资产

6月1日，施维雅宣布以26.5亿美元收购领先的肌肉病生物制药公司Edgewise Therapeutics（Nasdaq:EWTX），施维雅将以15.5亿美元的前期现金对价和高达11亿美元的额外里程碑付款收购sevasemten和Edgewise的肌肉营养不良业务。该交易有意义地加强了Edgewise的资产负债表，提高了财务灵活性，并突出了公司的战略重点，以加速和释放其心血管管道的全部潜力。交易完成后，Edgewise将成为一家专注于心血管疾病的公司，其产品线包括用于肥厚型心肌病的EDG-7500、用于HFpEF的EDG-15400和用于未披露靶点的EDG-003。 Sevasemten是一种研究性口服的一流快速骨骼肌球蛋白抑制剂，旨在保护和保护不稳定肌肉免受罕见肌营养不良患者收缩引起的损伤。Sevasemten目前在Becker肌营养不良症（BMD）的一个关键队列中进行了研究，并在Duchenne肌营养不良（DMD）的2期临床进行了研究。该候选药物有可能将施维雅定位为一家在神经肌肉疾病领域拥有强大能力和渠道的全球企业。根据协议条款，施维雅将获得sevasemten的所有权利，包括运营肌营养不良业务所需的相关知识产权、专有技术、关键协议、监管文件和临床数据。所有主要支持肌肉萎缩症业务的Edgewise员工都将在施维雅获得类似的报价，以确保发展的连续性和未来的商业执行。该交易反映了Edgewise的战略重点，即推进其心血管产品组合，同时将sevasemten定位于一家收购方，该收购方将带来充分实现sevasemten为患者的潜力所需的全球开发、监管和商业能力。 Edgewise therapeutics总裁兼首席执行官Kevin Koch博士表示：“施维雅专注于精准治疗，致力于治疗罕见的神经和神经肌肉疾病，使其成为该项目的理想管家。这项交易提供了即时的重大价值，包括15.5亿美元的预付款，同时将司维酮交给了一家拥有全球规模、患者承诺和商业影响力的收购方，以最大限度地发挥其对Becker和Duchenne肌营养不良症患者的潜力。同样重要的是，这项交易加强了我们的资产负债表，并提供了财务灵活性，以推动EDG-7500和EDG-15400通过关键的价值拐点。” 施维雅总裁Olivier Laureau表示：“Edgewise是肌肉疾病生物学的先驱，在为严重神经系统疾病患者发现和开发精确疗法方面有着良好的记录。收购sevasemten和Edgewise的肌营养不良业务，包括研究、开发、监管和商业职能方面经验丰富的人才，为我们将产品组合扩展到Becker和Duchenne肌营养不良症提供了一个直接的平台。根据我们2030年的目标，这使我们成为神经肌肉适应症的主要参与者，为更多患有毁灭性罕见疾病的患者提供服务。” 关注下方公众号，带你看世界!

并购临床2期

2026-06-01

·FierceBiotech

Servier inks $2.6B buyout of Edgewise’s muscular dystrophy unit to beef up neurology pipeline

Guggenheim analysts modeled a 55% probability of success for sevasemten in BMD. \n Servier has struck a deal to buy Edgewise Therapeutics’ muscular dystrophy business for $1.55 billion upfront, gaining control of a program that is on course to deliver pivotal data this year.Colorado-based Edgewise chose Becker muscular dystrophy (BMD) as the lead indication for sevasemten, an oral drug candidate designed to modulate the contraction of fast muscle fibers. Pivotal data in BMD, a rare genetic condition that causes progressive muscle weakness, are expected in the fourth quarter. Edgewise is also running a phase 2 trial of sevasemten in Duchenne muscular dystrophy (DMD).Edgewise’s pipeline features cardiovascular assets, including the midphase hypertrophic cardiomyopathy candidate EDG-7500. The Servier deal narrows Edgewise’s focus onto the cardiovascular pipeline while giving the biotech the cash to fuel development. Edgewise estimates the deal will fully fund EDG-7500 development through potential approval. In addition to the upfront fee, Servier has committed up to $1.1 billion in regulatory and commercial milestones to acquire Edgewise’s muscular dystrophy business. Sevasemten is Edgewise’s only molecule in clinical development as a treatment for muscular dystrophy. In a note to investors last week, Guggenheim Securities analysts modeled a 55% probability of success for sevasemten in BMD. The forecast reflected the mechanistic and functional proof-of-concept that Edgewise obtained through two clinical trials. In March, the biotech reported persistent stabilization of function for up to 3.5 years and no new safety concerns. After reviewing the data, Guggenheim analysts said they remained confident in a statistically significant outcome for Grand Canyon, the pivotal BMD cohort scheduled to deliver data this year. The evidence on sevasemten in DMD is less developed, leading the analysts to assign a 25% probability of success to the indication. Sevasemten has a different mechanism than DMD drugs such as Sarepta Therapeutics’ Exondys 51, which aim to boost levels of functional dystrophin. Rather than targeting the protein at the root of muscular dystrophies, sevasemten seeks to limit contraction of fast skeletal muscle fibers and protect them from injury and fibrosis. The mechanism could slow muscle breakdown and disease progression. Betting on the hypothesis ahead of pivotal data bolsters Servier’s neurology pipeline while exposing the French drugmaker to the risk of a late-phase failure. Neurology is one of Servier’s three pillars. However, Servier’s neurology pipeline is underdeveloped compared to the other pillars, with three clinical projects versus (PDF) 22 in oncology as of January. Expanding the neurology pipeline is a priority for Servier.

100 项与 Sevasemten 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
贝克型肌营养不良	临床3期	-	Edgewise Therapeutics, Inc.	-
糖原贮积病Ⅴ型	临床2期	丹麦	Edgewise Therapeutics, Inc.	2022-12-06
肢带型肌营养不良	临床2期	丹麦	Edgewise Therapeutics, Inc.	2022-12-06
杜氏肌营养不良症	临床2期	美国	Edgewise Therapeutics, Inc.	2022-10-24

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06066580 (MESA, NEWS) 人工标引	临床2期	贝克型肌营养不良	-	Sevasemten (ARCH trial participants)	觸鹹願製艱淵鹹醖鑰鹹(壓繭膚構鑰蓋鬱鏇築壓) = Sustained stabilization over 3.5 years: North Star Ambulatory Assessment (NSAA) functional scores of ARCH and CANYON trial participants treated with sevasemten remained stable after 3.5 years and 2 years, respectively. The CANYON participants on placebo who rolled into the MESA study also had NSAA functional scores that trended upward during the first year after switching to sevasemten. Once Becker muscular dystrophy functional decline begins, its course typically continues along a downward trajectory. Multiple natural history studies report NSAA scores declining by an average of 1.0 to 1.7 points annually for Becker patients, translating to an expected average functional drop of 3.0 to 5.1 points across 3 years. In the MESA extension study, sevasemten-treated patients showed NSAA outcomes that diverged markedly from predicted natural history declines: the CANYON cohort saw a slight NSAA improvement of +0.1 over 2 years versus a predicted natural decline of −2.9 points, while the ARCH cohort maintained a +0.1 NSAA benefit over 3.5 years compared with an expected historical decline of −5.3 points. 鏇蓋簾築夢餘憲構鹽廠 (獵窪鏇憲鬱襯鏇顧齋鬱 )	积极	2026-03-10
				Sevasemten (CANYON trial participants)
NCT05160415 (ARCH, MDA2026)	临床1期	贝克型肌营养不良	97	Sevasemten (Becker muscular dystrophy)	鑰構顧築蓋襯製鑰選淵(鏇範網夢觸鹽窪蓋襯簾) = 觸鹽選構蓋鏇觸糧餘衊蓋築廠夢衊蓋齋窪鑰鏇 (網網選膚觸鹽繭鏇願鑰, -0.4 ~ 0.8) 更多	积极	2026-03-08
NCT05160415 (ARCH, CTgov)	临床1期	贝克型肌营养不良	12	Sevasemten	鹽範範廠鹽窪餘醖獵襯 = 鑰膚淵壓窪艱鏇憲鏇壓衊壓壓餘餘齋選積鹹夢 (淵願選艱範獵築網襯壓, 積鏇夢衊製鑰憲選鬱蓋 ~ 夢簾獵構網艱範醖鬱願) 更多	-	2025-06-24
NCT05160415 (ARCH, MDA2025)	临床1期	贝克型肌营养不良	12	Sevasemten	憲簾範鹽衊鑰鏇鏇餘淵(鹹艱餘糧鏇醖窪鹹糧齋): Difference = 2.3 (95.0% CI, 1.0 ~ 3.6), P-Value = < 0.001 更多	积极	2025-03-16
				Sevasemten (Natural History Controls)
NCT05291091 (CANYON, MDA2025)	临床2期	贝克型肌营养不良	40	Sevasemten	艱積網鏇獵憲獵鹽膚壓(鏇鏇窪窪糧獵構醖壓範): P-Value = 0.02 更多	积极	2025-03-16
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NCT05291091 (CANYON, BusinessWire) 人工标引	临床2期	贝克型肌营养不良	-	Sevasemten	積鬱積鏇艱艱膚網襯顧(積膚鏇鹹遞鏇遞顧鏇範): Difference (%) = -28, P-Value = 0.02 达到更多	积极	2024-12-16
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NCT05160415 (ARCH, MDA2023) 人工标引	临床1期	杜氏肌营养不良症 CK \| TNNI2	12	EDG-5506 10-15 mg	顧窪壓遞衊積簾蓋齋構(顧壓壓製積範膚糧構夢) = 簾選獵膚夢鹹遞簾構糧夢蓋簾繭餘積獵淵膚網 (築顧鹹網鹹網獵觸廠餘 ) 更多	积极	2023-03-19
NCT05160415 (ARCH, Corporate Publications) 人工标引	临床1期	贝克型肌营养不良	12	EDG-5506	夢衊憲壓廠憲壓築醖製(蓋廠襯壓淵網鑰餘願築) = 25% Dizziness; 25% Somnolence; 25% Headache; 17% Fall; 8% Gastroenteritis virus 餘選願憲範構獵構鏇網 (鹽糧獵繭膚廠鬱願醖醖 )	积极	2022-09-12
NCT05160415 (ARCH, Corporate Publications) 人工标引	临床1期	贝克型肌营养不良	12	EDG-5506	糧艱築築衊範製遞餘繭(衊餘遞艱廠構觸選壓餘) = 糧糧窪繭淵製糧鑰網蓋繭廠選艱鏇網襯糧築獵 (糧餘壓範膚鏇製築糧製 ) 更多	积极	2022-06-20
NCT04585464 (BusinessWire) 人工标引	临床1期	贝克型肌营养不良	7	EDG-5506	網鹽願齋鹹遞襯積獵鏇(餘夢鬱艱壓蓋顧繭窪膚) = 願積膚廠築願願壓網觸築鬱蓋構鏇築觸鹹蓋積 (繭範窪鏇窪憲遞願積範 ) 更多	积极	2022-01-05
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